A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
1 other identifier
observational
353
10 countries
15
Brief Summary
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Oct 2008
Longer than P75 for all trials
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2008
CompletedFirst Submitted
Initial submission to the registry
November 6, 2008
CompletedFirst Posted
Study publicly available on registry
November 10, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2014
CompletedDecember 8, 2014
December 1, 2014
5.8 years
November 6, 2008
December 5, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Endurance
Study Visit
Respiratory Function
Study Visit
Study Arms (1)
MPS IVA
Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)
Eligibility Criteria
Subjects diagnosed with MPS IVA
You may qualify if:
- Willing and able to provide written, signed informed consent, or in the case of subjects age \< 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
- Willing and able to comply with all study procedures.
You may not qualify if:
- Use of any investigational product or investigational medical device within 30 days prior to screening.
- Previous hematopoietic stem cell transplant (HSCT).
- Concurrent disease or condition that would interfere with study participation or pose a safety concern.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
Unknown Facility
Oakland, California, United States
Unknown Facility
Chicago, Illinois, United States
Unknown Facility
Córdoba, Argentina
Unknown Facility
Porto Alegre, Brazil
Unknown Facility
São Paulo, Brazil
Unknown Facility
Montreal, Quebec, Canada
Unknown Facility
Lyon, France
Unknown Facility
Paris, France
Unknown Facility
Mainz, Germany
Unknown Facility
Monza, Italy
Unknown Facility
Amsterdam, Netherlands
Unknown Facility
Taipei, Taiwan
Unknown Facility
Birmingham, United Kingdom
Unknown Facility
London, United Kingdom
Unknown Facility
Manchester, United Kingdom
Related Publications (1)
Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.
PMID: 21877895DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Celeste Decker, MD
BioMarin Pharmaceutical
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 6, 2008
First Posted
November 10, 2008
Study Start
October 1, 2008
Primary Completion
July 1, 2014
Study Completion
July 1, 2014
Last Updated
December 8, 2014
Record last verified: 2014-12